• Cash Position: $257.2 million as of September 30, 2024, up from $236.2 million as of December 31, 2023.
• Collaboration Revenue: $0 in Q3 2024, down from $0.8 million in Q3 2023.
• Research and Development Expenses: $14.6 million in Q3 2024, down from $18.2 million in Q3 2023.
• General and Administrative Expenses: $8.4 million in Q3 2024, down from $10 million in Q3 2023.
• Net Loss: $21.7 million in Q3 2024, compared to $24 million in Q3 2023.
• Cash Guidance: Expected to end 2024 with approximately $240 million in cash, cash equivalents, and marketable securities.
• Cash Burn 2025: Projected to be approximately $55 million to $65 million.
• Funding Sufficiency: Existing cash expected to fund operations into at least 2027.

• Warning! GuruFocus has detected 2 Warning Signs with FULC.

Release Date: November 13, 2024

For the complete transcript of the earnings call, please refer to the full earnings call transcript.

Positive Points

• Fulcrum Therapeutics Inc (NASDAQ:FULC) has a strong cash position with $257.2 million in cash, cash equivalents, and marketable securities as of September 30, 2024.
• The company is advancing its pociredir program, an oral HbF inducer, for the treatment of sickle cell disease, with ongoing Phase 1b trials and plans to provide data in 2025.
• Fulcrum Therapeutics Inc (NASDAQ:FULC) is initiating Phase 1 clinical trials of pociredir in healthy volunteers, following positive interactions with the FDA.
• The company has a robust preclinical pipeline, including programs for inherited aplastic anemia, and plans to share additional information on development candidates soon.
• Fulcrum Therapeutics Inc (NASDAQ:FULC) has strengthened its leadership team with the addition of Rachel King to the Board of Directors and Dr. Thomas Winkler as Vice President of Hematology Clinical Development.

Negative Points

• The losmapimod program was suspended after failing to show separation from the placebo group in the Phase 3 REACH trial, leading to a 40% workforce reduction.
• There was no collaboration revenue in the third quarter of 2024, compared to $0.8 million in the same period of 2023, due to the completion of a collaboration agreement with MyoKardia.
• Research and development expenses decreased, primarily due to cost-sharing reimbursements, but this was partially offset by increased costs related to the advancement of the Phase 1b PIONEER trial.
• General and administrative expenses decreased due to workforce reductions, indicating potential challenges in maintaining operational efficiency.
• The net loss for the third quarter of 2024 was $21.7 million, slightly lower than the $24 million loss in the third quarter of 2023, but still significant.

Q & A Highlights

Q: How is Fulcrum Therapeutics approaching the decision to keep new assets wholly-owned versus partnering them out, and are there specific therapeutic areas of focus? A: Alex Sapir, CEO, explained that Fulcrum has a rich preclinical pipeline, with the most advanced program focused on inherited aplastic anemias. The company plans to progress these programs independently for now, with potential licensing opportunities for markets outside the US. The focus remains on progressing discovery efforts, particularly in inherited aplastic anemias.

Q: What feedback did Fulcrum receive from the FDA regarding the healthy volunteer studies for pociredir, especially in light of the OXBRYTA withdrawal? A: Alex Sapir noted that the FDA did not require additional clinical or preclinical data before proceeding with patient studies. The healthy volunteer studies are part of routine development, focusing on new formulations and drug interactions. Iain Fraser added that these studies are standard and unrelated to the OXBRYTA withdrawal.

Q: Can you provide guidance on how the data from the PIONEER trial for pociredir will be reported in 2025? A: Alex Sapir stated that the 12-milligram and 20-milligram cohorts will be reported separately at different times in 2025. The company is pleased with enrollment progress and plans to provide more detailed guidance on data sharing early in 2025. Patrick Horn added that the clinical development plan will be proposed to the FDA after the PIONEER study.

Q: How is Fulcrum using the experience with losmapimod to inform future programs, and how does it balance early discovery with external asset acquisition? A: Alex Sapir highlighted differences between losmapimod and pociredir, noting the strong evidence supporting pociredir's development. Fulcrum has a robust balance sheet to explore external assets but remains focused on exciting internal discovery programs. The company will be judicious and selective in any external acquisitions.

Q: What is the status of site activation for the PIONEER trial, and how many sites are expected to be activated? A: Alex Sapir confirmed that 12 sites are currently activated, with plans to reach 20 by the end of the year. Pat Horn added that the company has identified sites believed to be successful in recruiting for PIONEER, with ongoing activation efforts and potential for additional sites to join.

For the complete transcript of the earnings call, please refer to the full earnings call transcript.